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Table 1 Demographic and clinical variables in children with SMA

From: Assessment of motor function and nutritional status in children with spinal muscular atrophy treated with nusinersen after loading period in Western China: a retrospective study

 

SMA Type 1

SMA Type 2

SMA Type 3

Total

Number

8

31

7

46

Male/Female

3/5

14/17

3/4

20/26

SMN2 copy number

Two copies

1

5

0

6

Three copies

7

26

6

39

Positive family history

0

4

0

4

Age at symptom onset (y)

0.5 (0.33, 0.68)

0.9 (0.7, 1.3)

2.5 (1.3, 3.0)

0.95 (0.7, 1.3)

Age at genetic diagnosis (y)

0.65 (0.5, 1.18)

1.3 (1.0, 1.6)

2.9 (1.8, 3.7)

1.3 (0.875,1.85)

Disease duration (y)

0.15 (0.03, 0.2)

0.3 (0.1, 0.6)

0.5 (0.1, 0.7)

0.25 (0.08, 0.53)

Time from diagnosis to treatment (y)

2.05 (0.35, 4.7)

2.3 (0.8, 6.0)

1.2 (1.0, 4.1)

1.95 (0.8, 4.88)

Baseline age of functional assessment (y)

3.25 (2.19)

5.36 (4.09)

5.87 (3.19)

5.07 (3.74)

Ventilation

2

2

0

4

Tongue fasciculations

8

31

6

45

Areflexia/hyporeflexia

8

31

7

46

Dysphagia

2

0

0

2

Scoliosis

3

12

2

17

Arthrogryposis

2

12

0

14

Spinal surgery

0

1

0

1

Lumbar punctures in total

32

125

28

185

Baseline WAZ

−1.22 (1.5)

−0.52 (1.39)

−0.06 (0.80)

−0.59 (1.36)

Post-treatment WAZ

−1.51 (1.16)

− 1.1 (1.81)

− 0.67 (1.2)

−0.91(1.44)

P-value*

0.32

0.008

0.051

0.01

  1. SMA spinal muscular atrophy, SMN2 survival motor neuron 2, WAZ Weight /age Z-scores; Disease duration, a child’s age at genetic diagnosis minus the age at symptom onset; P-value*, P-value for baseline (M0), and post treatment (M2) WAZ